An alternative approach to endogenous gene-editing is the recombinant adeno-associated virus (rAAV) Genesis™ system from Horizon Discovery. Clearly differentiated from nuclease systems by not requiring DNA breaks, Genesis uses the cells’ natural homologous recombination machinery. Horizon claims its rAAV technology provides great flexibility in being able to sequence-mutate genes as easily as it can delete them, without causing the off-target alterations or sequence errors that can occur with nucleases, and with sensitivity at single base-pair resolution.
“While Horizon is relatively new to the commercial gene-editing arena, we are fast catching up. This is because of rAAV’s precision, but also because we are bringing significant advances in efficiency and throughput,” says Chris Torrance, Ph.D., CSO and founder of the company.
“We are also experts in translating this technology into better understanding gene function, how DNA mutations cause disease, and accelerating the process of novel target identification and drug discovery. Developing such tools and services is unique among gene-editing companies, but has allowed us to reach out to the wider research community, where it is still surprisingly underappreciated that gene editing is now an accessible and robust technology.”
Horizon has utilized Genesis to create more than 500 X-MAN™ (gene-X mutant and normal) genetically defined isogenic disease model cell lines. “These and many more disease models are needed to study cancer biology and support more efficient drug discovery, because genomics advances tell us that cancer is essentially hundreds of orphan diseases, comprised of multiple subtypes driven by distinct genetic features,” says Dr. Torrance.
“Cellular disease models (i.e., ‘patients-in-a-test-tube’) will greatly facilitate many aspects of novel targeted drug discovery, especially the stratification of patients into more focused clinical trials where they have the best chances of responding.
“Horizon is passionate about spreading the word on genome editing. We have already entered into many collaborations, including forming more than 30 centers of excellence with small and large nonprofit organizations, providing them free training in rAAV gene editing, and plan to launch ready-to-edit kits in the future. We are finding scientists very receptive to the technology, and we also get to expand our disease knowledge base and range of disease models. It is a win-win situation.”